Klingebiel Thomas, Boos Joachim, Beske Florian, Hallmen Erika, Int-Veen Christoph, Dantonello Tobias, Treuner Joern, Gadner Helmut, Marky Ildiko, Kazanowska Bernarda, Koscielniak Ewa
Children's Hospital of Frankfurt University Hospital, Frankfurt, Germany.
Pediatr Blood Cancer. 2008 Apr;50(4):739-45. doi: 10.1002/pbc.21494.
We prospectively studied the efficacy of high dose therapy (HDT) versus an oral maintenance treatment (OMT) in patients with stage IV soft tissue sarcoma (STS).
Both groups were pretreated with the CEVAIE combination consisting of carboplatin, etoposide, vincristine, actinomycin D, ifosfamide, and epirubicin. HDT consisted of a tandem cycle of thiotepa (600 mg/m(2)) plus cyclophosphamide (4,500 mg/m(2)) and melphalan (120 mg/m(2)) plus etoposide (1,800 mg/m(2)). This treatment was compared with OMT, consisting of four cycles trofosfamide (10 days 2 x 75 mg/m(2)/day) plus etoposide (10 days 2 x 25 mg/m(2)/day), and 4 cycles trofosfamide (10 days 2 x 75 mg/m(2)/day) plus idarubicin (10 days 4 x 5 mg/m(2)). Eligibility criteria were: diagnosis confirmed by reference pathology, primary stage IV, below 22 years of age, and having completed the study therapy.
From 96 patients 45 were treated with HDT and 51 with OMT. The main risk parameters were equally distributed in both arms. After a median follow-up of 57.4 months, 11/45 (24.4%) patients in the HDT-arm and 26/51 (57.8%) patients in OMT-arm were alive. Kaplan-Meier analysis demonstrated an overall survival for the whole group of 0.27 (OMT group: 0.52, HDT group 0.27, log rank P = 0.03). The proportional hazard analysis for patients with rhabdomyosarcoma (RMS) or "RMS-like" tumors (77.1% of all patients) demonstrated an independent benefit of OMT on outcome.
Oral maintenance therapy seems to be a promising option for patients with RMS-like stage IV tumors.
我们前瞻性地研究了高剂量疗法（HDT）与口服维持治疗（OMT）对IV期软组织肉瘤（STS）患者的疗效。
两组患者均先用由卡铂、依托泊苷、长春新碱、放线菌素D、异环磷酰胺和表柔比星组成的CEVAIE联合方案进行预处理。HDT包括硫替派（600mg/m²）加环磷酰胺（4500mg/m²）和马法兰（120mg/m²）加依托泊苷（1800mg/m²）的串联周期。该治疗方案与OMT进行比较，OMT包括四个周期的曲磷胺（10天，每天2次，每次75mg/m²）加依托泊苷（10天，每天2次，每次25mg/m²），以及四个周期的曲磷胺（10天，每天2次，每次75mg/m²）加伊达比星（10天，每天4次，每次5mg/m²）。入选标准为：经参考病理确诊、原发IV期、年龄在22岁以下且已完成研究治疗。
96例患者中，45例接受HDT治疗，51例接受OMT治疗。主要风险参数在两组中分布均衡。中位随访57.4个月后，HDT组有11/45（24.4%）的患者存活，OMT组有26/51（57.8%）的患者存活。Kaplan-Meier分析显示，全组的总生存率为0.27（OMT组：0.52，HDT组：0.27，对数秩检验P = 0.03）。对横纹肌肉瘤（RMS）或“RMS样”肿瘤患者（占所有患者的77.1%）进行的比例风险分析显示，OMT对预后有独立的益处。
对于IV期RMS样肿瘤患者，口服维持治疗似乎是一个有前景的选择。
